PMID- 28951660
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20201001
IS  - 1179-5468 (Print)
IS  - 1179-5468 (Electronic)
IS  - 1179-5468 (Linking)
VI  - 11
DP  - 2017
TI  - Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin 
      Cardiomyopathy.
PG  - 1179546817730322
LID - 10.1177/1179546817730322 [doi]
LID - 1179546817730322
AB  - BACKGROUND: Transthyretin cardiomyopathy (TTR-CM) is a progressive, fatal disease 
      caused by the accumulation of misfolded transthyretin (TTR) amyloid fibrils in 
      the heart. Tafamidis is a kinetic stabilizer of TTR that inhibits misfolding and 
      amyloid formation. METHODS: In this post hoc analysis, data from an observational 
      study (Transthyretin Amyloidosis Cardiac Study; n = 29) were compared with an 
      open-label study of tafamidis in patients with TTR-CM (Fx1B-201; n = 35). To 
      ensure comparable baseline disease severity, patients with New York Heart 
      Association (NYHA) functional classification >/=III were excluded in this 
      time-to-mortality analysis. RESULTS: Patients with either wild-type or Val122Ile 
      genotypes treated with tafamidis have a significantly longer time to death 
      compared with untreated patients (P = .0004). Similar results were obtained when 
      limiting the analysis to wild-type patients only, without restricting NYHA 
      functional classification (P = .0262). CONCLUSIONS: These results support earlier 
      conclusions suggesting that tafamidis slows disease progression compared with no 
      treatment outside of standard of care and warrant further investigation. TRIAL 
      REGISTRATION: ClinicalTrials.gov, NCT00694161.
FAU - Sultan, Marla B
AU  - Sultan MB
AD  - Global Product Development, Pfizer Inc, New York, NY, USA.
FAU - Gundapaneni, Balarama
AU  - Gundapaneni B
AD  - Biostatistics, inVentiv Health, Burlington, MA, USA.
FAU - Schumacher, Jennifer
AU  - Schumacher J
AD  - Global Medical Affairs, Pfizer Inc, New York, NY, USA.
FAU - Schwartz, Jeffrey H
AU  - Schwartz JH
AD  - Global Product Development, Pfizer Inc, New York, NY, USA.
LA  - eng
SI  - ClinicalTrials.gov/NCT00694161
PT  - Journal Article
DEP - 20170918
PL  - United States
TA  - Clin Med Insights Cardiol
JT  - Clinical Medicine Insights. Cardiology
JID - 101525768
PMC - PMC5606341
OTO - NOTNLM
OT  - Transthyretin
OT  - cardiac amyloidosis
OT  - heart failure
OT  - tafamidis
COIS- Declaration of conflicting interests:The author(s) declared the following 
      potential conflicts of interest with respect to the research, authorship, and/or 
      publication of this article: M.B.S., J.S., and J.H.S. are full-time employees of 
      Pfizer and hold stock and/or stock options. B.G. is a full-time employee of 
      inVentiv Health, a paid contractor to Pfizer for statistical analysis and 
      development of this manuscript.
EDAT- 2017/09/28 06:00
MHDA- 2017/09/28 06:01
PMCR- 2017/09/18
CRDT- 2017/09/28 06:00
PHST- 2017/03/29 00:00 [received]
PHST- 2017/08/15 00:00 [accepted]
PHST- 2017/09/28 06:00 [entrez]
PHST- 2017/09/28 06:00 [pubmed]
PHST- 2017/09/28 06:01 [medline]
PHST- 2017/09/18 00:00 [pmc-release]
AID - 10.1177_1179546817730322 [pii]
AID - 10.1177/1179546817730322 [doi]
PST - epublish
SO  - Clin Med Insights Cardiol. 2017 Sep 18;11:1179546817730322. doi: 
      10.1177/1179546817730322. eCollection 2017.